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Beam Therapeutics to Present First Research Highlighting Approach to Develop Non-Genotoxic Conditioning Regimens for Patients with Sickle Cell Disease Ahead of Autologous Transplant
CAMBRIDGE, Mass., June 27, 2022 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines
About this update from Beam Therapeutics Inc.
[{"type":"text","content":"CAMBRIDGE, Mass., June 27, 2022 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that new research highlighting the company’s internal efforts to develop improved transplant conditioning regimens for patients with sickle cell disease (SCD) undergoing hematopoietic stem cell transplantation (HSCT) will be presented today, June 27, 2022, at 4:00 p.m. CEST at the Federation of American Societies for Experimental Biology (FASEB) Genome Engineering Conference by Nicole Gaudelli, Ph.D., director, head of gene editing at Beam. The conference is being held from June 26-30, 2022, in Lisbon, Portugal. Beam is advancing two ex vivo base editing programs for SCD: BEAM-101, which incorporates base edits that are designed to mimic single nucleotide polymorphisms seen in individuals with hereditary persistence of fetal hemoglobin, and BEAM-102, which directly edits the causative HbS point mutation to recreate a naturally occurring normal human hemoglobin variant, HbG-Makassar. In the second half of 2022, Beam plans to initiate patient enrollment in its clinical trial with BEAM-101 and to submit an investigational new drug application for BEAM-102. Beam has laid out a long-term strategy to support broad accessibility of base editing treatments for patients with SCD and other hematologic diseases. A key component of this strategy is focused on improving the safety of conditioning regimens, a required pretreatment for patients receiving ex vivo gene editing treatment via autologous transplant. Today’s conditioning regimens rely on nonspecific chemotherapy or radiation, which are associated with significant toxicities, including genotoxicity, primary or secondary malignancy, and organ toxicities including infertility. With a goal of overcoming this, Beam has leveraged its base editing capabilities to develop a potentially non-genotoxic approach that combines antibody-based conditioning with multiplex gene edited hematopoietic stem cells (HSCs) called ESCAPE, or Engineered Stem Cell Antibody Paired Evasion. “As we execute on our long-term strategy to develop base editing treatments for SCD, we are excited to share new findings around our pre-clinical research to identify improved conditioning regimens for patients ahead of autologous trans...